Abstract | BACKGROUND: METHODS: We studied 14 patients with severe osteopetrosis treated with subcutaneous injections of recombinant human interferon gamma-1b (1.5 micrograms per kilogram of body weight per dose) three times per week for at least 6 months; 11 patients were treated for 18 months. We assessed the effect of therapy by evaluating the patients' clinical status, measuring blood counts and biochemical markers of bone turnover, and performing bone marrow imaging and bone biopsies. RESULTS: After 6 months of therapy, all 14 patients had decreases in trabecular-bone area (determined by histomorphometric analysis of bone-biopsy specimens) and increases in bone marrow space (determined by marrow imaging), and the improvement was sustained in the 11 patients treated for 18 months. The mean (+SD) hemoglobin concentration increased from 7.5 +/- 2.9 to 10.5 +/- 0.3 g per deciliter (P = 0.05), and superoxide generation by granulocyte-macrophage colonies increased (P < 0.001) after 18 months of therapy. In six patients for whom pretreatment data were available, there was a 96 percent decrease in the frequency of infections requiring antibiotic therapy during interferon treatment. There were no side effects necessitating the discontinuation of therapy. CONCLUSIONS:
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Authors | L L Key Jr, R M Rodriguiz, S M Willi, N M Wright, H C Hatcher, D R Eyre, J K Cure, P P Griffin, W L Ries |
Journal | The New England journal of medicine
(N Engl J Med)
Vol. 332
Issue 24
Pg. 1594-9
(Jun 15 1995)
ISSN: 0028-4793 [Print] United States |
PMID | 7753137
(Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)
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Chemical References |
- Recombinant Proteins
- Interferon-gamma
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Topics |
- Adult
- Bone Resorption
- Child, Preschool
- Humans
- Injections, Subcutaneous
- Interferon-gamma
(administration & dosage, therapeutic use)
- Osteopetrosis
(congenital, pathology, therapy)
- Recombinant Proteins
(administration & dosage, therapeutic use)
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